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This review of the French health system analyses recent developments in health organisation and governance, financing, healthcare provision, recent reforms and health system performance. Overall health status continues to improve in France, although geographic and socioeconomic inequalities in life expectancy persist. The health system combines a social health insurance (SHI) model with an important role for tax-based revenues to finance healthcare. The health system provides universal coverage, with a broad benefits basket, but cost-sharing is required for all essential services. Private complementary insurance to cover these costs results in very low average out-of-pocket (OOP) payments, although there are concerns regarding solidarity, financial redistribution and efficiency in the health system. The macroeconomic context in the last couple of years in the country has been affected by the Covid-19 pandemic, which resulted in subsequent increases of total health expenditure in France in 2020 (3.7%) and 2021 (9.8%). Healthcare provision continues to be highly fragmented in France, with a segmented approach to care organization and funding across primary, secondary and long-term care. Recent reforms aim to strengthen primary care by encouraging multidisciplinary group practices, while public health efforts over the last decade have focused on boosting prevention strategies and tackling lifestyle risk factors, such as smoking and obesity with limited success. Continued challenges include ensuring the sustainability of the health workforce, particularly to secure adequate numbers of health professionals in medically underserved areas, such as rural and less affluent communities, and improving working conditions, remuneration and career prospects, especially for nurses, to support retention. The Covid-19 pandemic has brought to light some structural weaknesses within the French health system, but it has also provided opportunities for improving its sustainability. There has been a notable shift in the will to give more room to decision-making at the local level, involving healthcare professionals, and to find new ways of funding healthcare providers to encourage care coordination and integration., (World Health Organization 2023 (acting as the host organization for, and secretariat of, the European Observatory on Health Systems and Policies).)

With over one million deaths per year in the world, suicide is a major public health problem that could be significantly reduced by effective prevention programs. E-health tools are of particular interest for primary prevention as they can address a broad population including people unaware of their own risk and provide information and help without the fear of stigma. Our main objective was to define the overall characteristics of an e-health tool for suicide primary prevention in the French general population by defining the characteristics of the IT features; the content of the information delivered; the best way to structure it; and how it should be relayed and by whom. The research was carried out through a literature review and a co-construction phase with stakeholders. Four types of strategies may guide the construction of e-health tools for suicide primary prevention: education and awareness, (self-)screening, accessing support, and mental health coping. They should be accessible on different devices to reach the most users, and language and content should be adapted to the target population and to the issue being addressed. Finally, the tool should be consistent with ethical and quality best practices. The e-health tool StopBlues was developed following those recommendations.

Background: HPV vaccine coverage in France remained lower than in most other high-income countries. Within the diagnostic phase of the national PrevHPV program, we carried out a mixed methods study among school staff to assess their knowledge, beliefs and attitudes regarding HPV, HPV vaccine and vaccination in general, and regarding schools' role in promoting HPV vaccination., Methods: Middle school nurses, teachers and support staff from four French regions participated between January 2020 and May 2021. We combined: (i) quantitative data from self-administered online questionnaires (n = 301), analysed using descriptive statistics; and (ii) qualitative data from three focus groups (n = 14), thematically analysed., Results: Less than half of respondents knew that HPV can cause genital warts or oral cancers and only 18% that no antiviral treatment exists. Almost 90% of the respondents knew the existence of the HPV vaccine but some misunderstood why it is recommended before the first sexual relationships and for boys; 56% doubted about its safety, especially because they think there is not enough information on this topic. Schools nurses had greater knowledge than other professionals and claimed that educating pupils about HPV was fully part of their job roles; however, they rarely address this topic due to a lack of knowledge/tools. Professionals (school nurses, teachers and support staff) who participated in the focus groups were unfavourable to offering vaccination at school because of parents' negative reactions, lack of resources, and perceived uselessness., Conclusions: These results highlight the need to improve school staff knowledge on HPV. Parents should be involved in intervention promoting HPV vaccination to prevent their potential negative reactions, as feared by school staff. Several barriers should also be addressed before organizing school vaccination programs in France., (© 2023. The Author(s).)

Background: The social protection scheme in charge of farmers and agricultural employees (MSA) in France has developed a two-step health promotion program with a nurse appointment followed by a consultation with a doctor of the participant's choosing to reach its under-consuming beneficiaries and enroll them back into a care pathway. Our objective was to carry out a pilot evaluation of this program., Methods: The evaluation was carried out on the population invited during the second semester of 2017 using data from the program's service providers (date of invitation, of nurse appointment…), regional MSA bodies (consultation voucher), and reimbursement data (other care consumption). Participation rates were calculated overall and by participant characteristics. Medical needs were identified during the nurse appointment and new care pathways were assessed using reimbursement data. Multivariable regression models identified factors associated with participation., Results: 2366 beneficiaries were included in the analysis. 1559 (65.89%) were men and mean age was 52.41 (standard deviation = 14.86). 409 (17.29%) attended the nurse appointment. There was a significant increase in participation with age, in farmers vs. employees (odds ratio = 1.905, 95% confidence interval = 1.393-2.604), and in people living in the most disadvantaged areas (odds ratio = 1.579, 95% confidence interval=1.079-2.312). Participation to the consultation following the nurse appointment was high (62.35%-73.11%). 87.53% of participants had at least one medical need, and new care pathways were more frequent among those who had attended the nurse appointment (55.50% vs. 34.80%, p < 0.0001)., Conclusions: This pilot evaluation shows promising results which need to be confirmed with a national evaluation of the program and longer-term evidence., Competing Interests: Conflict of interest statement MM, CD and KC report no conflict of interest apart from the funding received to carry out the study. MAS and VD are both employees of the funder, the Mutualité Sociale Agricole central body (CCMSA, Caisse centrale de la Mutualité Sociale Agricole)., (Copyright © 2022 Elsevier Masson SAS. All rights reserved.)

Objective: To look at the association of socioeconomic status (SES) with the suitability of the maternity where children are born and its association with mortality., Design: Retrospective analysis of a prospective cohort constituted using hospital discharge databases., Setting: France POPULATION: Live births in 2012-2014 in maternity hospitals in mainland France followed until discharge from the hospital., Main Outcome Measure: Unsuitability of the maternity to newborns' needs based on birth weight and gestational age, early transfers (within 24 hours of birth) and in-hospital mortality., Results: 2 149 454 births were included, among which 155 646 (7.2%) were preterm. Preterm newborns with low SES were less frequently born in level III maternities than those with high SES. They had higher odds of being born in an unsuitable maternity (OR=1.174, 95% CI 1.114 to 1.238 in the lowest SES quintile compared with the highest), and no increase in the odds of an early transfer (OR=0.966, 95% CI 0.849 to 1.099 in the lowest SES quintile compared with the highest). Overall, newborns from the lowest SES quintile had a 40% increase in their odds of dying compared with the highest (OR=1.399, 95% CI 1.235 to 1.584)., Conclusions: Newborns with the lowest SES were less likely to be born in level III maternity hospitals compared with those with the highest SES, despite having higher prematurity rates. This was associated with a significantly higher mortality in newborns with the lowest SES. Strategies must be developed to increase health equity among mothers and newborns., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2022. No commercial re-use. See rights and permissions. Published by BMJ.)

Background: To identify deprivation indicators usable in everyday practice and included in medico-administrative databases, particularly with infectious diseases, which represent the greatest proportion of hospitalizations. Our objective was to compare ecological indicators to individual questionnaires and apply both types to the study of the impact of deprivation on hospital efficiency., Methods: We conducted an epidemiological observational prospective multicentre study in two French public hospitals between 20 October 2016 and 20 March 2017. Children hospitalized for one of the four most common infectious diseases were included and their parents were asked to answer the Evaluation of Precarity and Inequalities in Health Examination Centers (EPICES) questionnaire. The ecological indicator French DEPrivation index (FDep) was derived from patients' address, both at the zip code and at a smaller geographical area (IRIS [ilôts de regroupement pour l'information statistique]) level. Correlation and concordance between the three indicators were assessed. The endpoint used to assess the impact on hospital efficiency was the ratio between patients' length of stay (LOS) and the national LOS of their disease-related group., Results: Data were available for 540 patients with a mean age of 9 mo. A total of 56.1% of patients were considered deprived with EPICES, 50.4% with zip code FDep and 45.7% with IRIS FDep. Concordance between EPICES and either type of FDep was <0.1. There was no increase in LOS compared with national LOS with any of the indicators., Conclusions: Individual and ecological indicators do not measure the same aspects of deprivation. The decision to use one or the other must be carefully weighed when studying the impact of deprivation on the healthcare system., (© The Author(s) 2020. Published by Oxford University Press on behalf of Royal Society of Tropical Medicine and Hygiene.)

Background: For over a decade, digital health has held promise for enabling broader access to health information, education, and services for the general population at a lower cost. However, recent studies have shown mixed results leading to a certain disappointment regarding the benefits of eHealth technologies. In this context, community-based health promotion represents an interesting and efficient conceptual framework that could help increase the adoption of digital health solutions and facilitate their evaluation., Objective: To understand how the local implementation of the promotion of an eHealth tool, StopBlues (SB), aimed at preventing psychological distress and suicide, varied according to local contexts and if the implementation was related to the use of the tool., Methods: The study was nested within a cluster-randomized controlled trial that was conducted to evaluate the effectiveness of the promotion, with before and after observation (NCT03565562). Data from questionnaires, observations, and institutional sources were collected in 27 localities where SB was implemented. A multiple correspondence analysis was performed to assess the relations between context, type of implementation and promotion, and use of the tool., Results: Three distinct promotion patterns emerged according to the profiles of the localities that were associated with specific SB utilization rates. From highest to lowest utilization rates, they are listed as follows: the privileged urban localities, investing in health that implemented a high-intensity and digital promotion, demonstrating a greater capacity to take ownership of the project; the urban, but less privileged localities that, in spite of having relatively little experience in health policy implementation, managed to implement a traditional and high-intensity promotion; and the rural localities, with little experience in addressing health issues, that implemented low-intensity promotion but could not overcome the challenges associated with their local context., Conclusions: These findings indicate the substantial influence of local context on the reception of digital tools. The urban and socioeconomic status profiles of the localities, along with their investment and pre-existing experience in health, appear to be critical for shaping the promotion and implementation of eHealth tools in terms of intensity and use of digital communication. The more digital channels used, the higher the utilization rates, ultimately leading to the overall success of the intervention., International Registered Report Identifier (irrid): RR2-10.1186/s13063-020-04464-2., (©Kathleen Turmaine, Agnès Dumas, Karine Chevreul, PRINTEMPS Consortium. Originally published in the Journal of Medical Internet Research (https://www.jmir.org), 22.04.2022.)

Introduction: Vaccination is an effective and safe strategy to prevent Human papillomavirus (HPV) infection and related harms. Despite various efforts by French authorities to improve HPV vaccine coverage (VC) these past few years, VC has remained far lower than in most other high-income countries. To improve it, we have coconstructed with stakeholders a school-based and primary care-based multicomponent intervention, and plan to evaluate its effectiveness, efficiency and implementation through a cluster randomised controlled trial (cRCT)., Methods and Analysis: This pragmatic cRCT uses an incomplete factorial design to evaluate three components applied alone or in combination: (1) adolescents and parents' education and motivation at school, using eHealth tools and participatory learning; (2) general practitioners' training on HPV using motivational interviewing techniques and provision of a decision aid tool; (3) free-of-charge access to vaccination at school. Eligible municipalities (clusters) are located in one of 14 preselected French school districts and must have only one secondary school which enrols at least 2/3 of inhabitants aged 11-14 years. A randomisation stratified by school district and deprivation index allocated 90 municipalities into 6 groups of 15. The expected overall sample size estimate is 41 940 adolescents aged 11-14 years. The primary endpoint is the HPV VC (≥1 dose) among adolescents aged 11-14 years, at 2 months, at the municipality level (data from routine databases). Secondary endpoints include: HPV VC (≥1 dose at 6 and 12 months; and 2 doses at 2, 6 and 12 months); differences in knowledge, attitudes, behaviours, and intention among adolescents, parents and general practitioners between baseline and 2 months after intervention (self-administered questionnaires); incremental cost-effectiveness ratio. Implementation measures include dose, fidelity, adaptations, reached population and satisfaction (activity reports and self-administered questionnaires)., Ethics and Dissemination: This protocol was approved by the French Ethics Committee 'CPP Sud-Est VI' on 22 December 2020 (ID-RCB: 2020-A02031-38). No individual consent was required for this type of research; all participants were informed of their rights, in particular not to participate or to oppose the collection of data concerning them. Findings will be widely disseminated (conference presentations, reports, factsheets and academic publications)., Trial Registration Number: NCT04945655., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2022. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Aims: Transcatheter aortic valve replacement (TAVR) as an alternative to surgical aortic valve replacement (SAVR) has profoundly changed the management of patients with aortic valve stenosis (AS). Large unbiased nationwide data regarding TAVR implementation, impact on SAVR and their respective outcomes are scarce., Methods and Results: Based on a French administrative hospital-discharge database, we collected data on all consecutive aortic valve replacements (AVRs) performed in France for AS between 2007 and 2019 [106 253 isolated SAVR (49%), 46 514 combined SAVR (21%), and 65 651 TAVR (30%)]. The number of AVR linearly increased between 2007 and 2019 (from 10 892 to 23 109, P for trend < 0.0001) due to a marked increase in TAVR (from 253 to 13 030, P for trend < 0.0001), while SAVR increased up to 2013 and then declined (10 892 in 2007, 12 699 in 2013, and 10 079 in 2019). The Charlson index decreased linearly for TAVR, but in two steps for SAVR (2011 and 2017). In-hospital mortality rates of both SAVR and TAVR declined (both P for trend < 0.0001) and were similar or lower for TAVR than for isolated SAVR in patients 75 years or above in the last 3 years (2017-19). Complication rates of TAVR also declined but permanent pacemaker rates remained high and length of stay substantial (16.7% and median 6 days, respectively, in 2017-19)., Conclusion: The number of AVR has doubled in a decade and TAVR has become the dominant form of AVR in 2018. The improvement in patient profiles seems to have anticipated the demonstrated benefit of TAVR in intermediate and low-risk patients. In patients 75 years or older, TAVR should be considered as the first option. We also highlight two important areas for improvement, the high permanent pacemaker rates, and the long length of stay even in the contemporary era. Our results may have major implications for clinical practice and policymakers., (Published on behalf of the European Society of Cardiology. All rights reserved. © The Author(s) 2021. For permissions, please email: journals.permissions@oup.com.)

Objective: This study looks at tobacco use among persons with psychiatric disorders and its management in public psychiatric units in France., Methods: A thematic analysis based on semi-directive interviews with 10 patients and 9 caregivers was carried out., Results: Patients use tobacco to deal with their emotions and mood swings. However, mental health professionals, who only focus on mental disorders and addictions that have a direct impact on behaviors, disregard smoking addiction. Moreover, tobacco is sometimes integrated into the way services operate. However, it would be too simplistic to reduce the issue of smoking cessation to a matter of goodwill of caregivers. The structural dimension of smoking was evident, particularly in psychiatric hospitals, where it was an important bargaining chip and activity., Conclusions: Patients desire to receive individualized smoking-cessation interventions. This underlines the relevance of a public health intervention co-construction that goes beyond simply prescribing nicotine substitutes. This intervention should take into account characteristics of tobacco consumption among psychiatric patients, and the need to offer such interventions when patients are stabilized. In the meantime, psychiatric caregivers should be encouraged to engage in shared decision-making processes with patients for tobacco-use reduction and cessation.

Objectives: This study aimed to evaluate the uncertainty related to the use of common collection tools to assess costs in economic evaluations compared with an exhaustive administrative database., Methods: A pragmatic study was performed using preexisting cost-effectiveness studies. Patients were probabilistically matched with themselves in the French National Health Data System (Système National des Données de Santé [SNDS]), and all their reimbursed hospital and ambulatory care data during the study were extracted. Outcomes included the ratio of the number of each type of resources consumed using trial data (case report forms for ambulatory care and local hospital data for hospital care) versus the SNDS and the ratio of corresponding costs. Mean ratios and 95% confidence intervals (CIs) were calculated using bootstrapping. The impact of the collection tool on the result of the economic evaluation was calculated with the difference in costs between the 2 treatment arms with both collection methods., Results: Five cost-effectiveness studies were included in the analysis. A total of 397 patients had the SNDS hospital data, and 321 had ambulatory care data. Common collection tools underestimated hospital admissions by 13% (95% CI 8-20), corresponding costs by 5% (95% CI 2-14), and ambulatory acts by 41% (95% CI 33-51), with large variations in costs depending on the study. There was no change in the economic conclusion in any study., Conclusions: The use of common collection tools underestimates healthcare resource consumption and its associated costs, particularly for ambulatory care. Our results could provide useful evidence-based estimates to inform sensitivity analyses' parameters in future cost-effectiveness analyses., (Copyright © 2021 ISPOR–The Professional Society for Health Economics and Outcomes Research. Published by Elsevier Inc. All rights reserved.)

Background: According to the guidelines, surgical aortic valve replacement (SAVR) is recommended in patients at low surgical risk (EuroSCORE II<4%), whereas for other patients, the decision between transcatheter aortic valve implantation (TAVI) and surgery should be made by the Heart Team, with TAVI being favoured in elderly patients., Aim: The RAC prospective multicentre survey assessed the respective contributions of age and surgical risk scores in therapeutic decision making in elderly patients with severe symptomatic aortic stenosis., Methods: In September and October 2016, 1049 consecutive patients aged ≥ 75 years were included in 32 centres with on-site TAVI and surgical facilities. The primary endpoint was the decision between medical management, TAVI or SAVR., Results: Mean age was 84±5 years and 53% of patients were female. The surgical risk was classified as high (EuroSCORE II>8%) in 18% of patients, intermediate (EuroSCORE II 4-8%) in 34% and low (EuroSCORE II≤4%) in 48%. TAVI was preferred in 71% of patients, SAVR in 19% and medical treatment in 10%. The choice of TAVI over SAVR was associated with older age (P<0.0001) and a higher EuroSCORE II (P=0.008). However, the weight of EuroSCORE II in therapeutic decision making markedly decreased after the age of 80 years. Indeed, 77% of patients aged ≥ 80 years were referred for TAVI, despite a low estimated surgical risk., Conclusions: The impact of risk scores depends strongly on age, and decreases considerably after 80 years, most patients being referred for TAVI, independent of their estimated surgical risk. Despite medical advancements, 10% of patients were still denied any intervention., (Copyright © 2021 Elsevier Masson SAS. All rights reserved.)

Background: Despite the high prevalence of child abuse and neglect and its consequences on health, very few studies have evaluated the trajectories of children in placement in France, and there are no cost studies available., Objective: To estimate the cost of abuse and neglect in children in placement before their 4th birthday in France., Participants and Setting: A cohort of children in placement before their 4th birthday in a single nursery between February 1994 and June 2001 was followed from birth until they left care., Methods: Child protective services (CPS), health, health and social, and judiciary services utilization was derived from a qualitative analysis of the children's files and valued with their unit costs in 2013 euros from the societal perspective. Total costs and mean annual cost per child followed by CPS were calculated overall, by cost category, and by status at admission to the nursery., Results: 129 children were included. Mean age at first admission was 1.9 years (SD = 1.3). The mean length of follow-up by CPS was 14.3 years (SD = 5.0). Mean annual cost per child was estimated at €53,265 (SD = 42,077), with CPS costs representing 78% of costs. 80% of health care costs were due to psychiatric care. Children with no identified psychosocial risk factors had significantly higher psychiatric care costs and health and social care costs than pre-term children and children with identified psychosocial risk factors., Conclusion: More research should be carried out to address early and comprehensively the multiple needs of children followed by CPS in the short- and long-term., (Copyright © 2021 Elsevier Ltd. All rights reserved.)

Ed-LinQ is a mental health policy initiative to enhance the early detection and treatment of children with mental illness by improving the liaison between schools and health services in Queensland, Australia. We measured its impact from policy to practice to inform further program developments and public strategies. We followed a mixed quantitative/qualitative approach. The Adoption Impact Ladder (AIL) was used to analyse the adoption of this initiative by end-users (decision makers both in the health and education sectors) and the penetration of the initiative in the school sector. Survey respondents included representatives of schools ( n = 186) and mental health providers ( n = 78). In total, 63% of the school representative respondents were at least aware of the existence of the Ed-LinQ initiative, 74% were satisfied with the initiative and 28% of the respondent schools adopted the initiative to a significant extent. Adoption was higher in urban districts and in the health sector. The overall level of penetration in the school sector of Queensland was low (3%). The qualitative analysis indicated an improvement in the referral and communication processes between schools and the health sectors and the importance of funding in the implementation of the initiative. Mapping of existing programs is needed to assess the implementation of a new one as well as the design of different implementation strategies for urban and rural areas. Assessing the adoption of health policy strategies and their penetration in a target audience is critical to understand their proportional impacts across a defined ecosystem and constitutes a necessary preliminary step for the evaluation of their quality and efficiency.

Background: Suicide constitutes a cause of death which could be prevented by e-health programs accessible to the general population. Effective promotion has the potential to maximize the uptake of such programs. However, few e-health programs have been combined with promotion campaigns. The primary objective of this trial is to assess the effectiveness of a tailored promotion, at a local level, of a mobile application and website offering evidence-based content for suicide prevention (the StopBlues program), and to compare the effectiveness of two types of local promotion in terms of their impact on suicidal acts. Secondary objectives focus on the effectiveness of the promotion in terms of the intensity of utilization of the StopBlues program, help-seeking behaviors and the level of psychological impairment of program users., Methods/design: This is a three-arm, parallel-group, cluster-randomized controlled trial, with before-and-after observation. Thirty-four clusters, corresponding to geographical areas sharing a common local authority in France, will be included. They will be randomly assigned to one of the following arms with a ratio of 1:1:1: a control group; a basic promotion group in which promotion of the StopBlues program will be done by local authorities; and an intensified promotion group in which basic promotion will be supplemented by an additional one in a general practitioner's waiting room. The primary outcome measure will be the number of suicidal acts within each cluster over a 12-month period following the launch of the intervention. Baseline data will be collected for each cluster over the 12-month period prior to the trial. Secondary outcomes will include length of use of the StopBlues program, measures of help-seeking behaviors and level of psychological distress among users of the program, as well as the cost-effectiveness and budgetary impact of its promotion. A more sustained promotion by local authorities will also be implemented after 12 months in the control group and assessed using the same outcome measures., Discussion: This research should contribute to the sparse evidence base regarding the promotion of e-health programs and will support the wider delivery of the intervention evaluated if proven effective., Trial Registration: ClinicalTrials.gov, ID: NCT03565562. Registered on 11 June 2018.

Objectives: Studies have shown a consistent impact of socioeconomic status at birth for both mother and child; however, no study has looked at its impact on hospital efficiency and financial balance at birth, which could be major if newborns from disadvantaged families have an average length of stay (LOS) longer than other newborns. Our objective was therefore to study the association between socioeconomic status and hospital efficiency and financial balance in that population., Methods: A study was carried out using exhaustive national hospital discharge databases. All live births in a maternity hospital located in mainland France between 2012 and 2014 were included. Socioeconomic status was estimated with an ecological indicator and efficiency by variations in patient LOS compared with different mean national LOS. Financial balance was assessed at the admission level through the ratio of production costs and revenues and at the hospital level by the difference in aggregated revenues and production costs for said hospital. Multivariate regression models studied the association between those indicators and socioeconomic status., Results: A total of 2 149 454 births were included. LOS was shorter than the national means for less disadvantaged patients and longer for the more disadvantaged patients, which increased when adjusted for gestational age, birth weight, and severity. A 1% increase in disadvantaged patients in a hospital's case mix significantly increased the probability that the hospital would be in deficit by 2.6%., Conclusions: Reforms should be made to hospital payment methods to take into account patient socioeconomic status so as to improve resource allocation efficiency., (Copyright © 2019 ISPOR–The Professional Society for Health Economics and Outcomes Research. Published by Elsevier Inc. All rights reserved.)

A variety of effective psychotherapies for depression are available, but patients who suffer from depression vary in their treatment response. Combining face-to-face therapies with internet-based elements in the sense of blended treatment is a new approach to treatment for depression. The goal of this study was to answer the following research questions: (1) What are the most important predictors determining optimal treatment allocation to treatment as usual or blended treatment? and (2) Would model-determined treatment allocation using this predictive information and the personalized advantage index (PAI)-approach result in better treatment outcomes? Bayesian model averaging (BMA) was applied to the data of a randomized controlled trial (RCT) comparing the efficacy of treatment as usual and blended treatment in depressive outpatients. Pre-treatment symptomatology and treatment expectancy predicted outcomes irrespective of treatment condition, whereas different prescriptive predictors were found. A PAI of 2.33 PHQ-9 points was found, meaning that patients who would have received the treatment that is optimal for them would have had a post-treatment PHQ-9 score that is two points lower than if they had received the treatment that is suboptimal for them. For 29% of the sample, the PAI was five or greater, which means that a substantial difference between the two treatments was predicted. The use of the PAI approach for clinical practice must be further confirmed in prospective research; the current study supports the identification of specific interventions favorable for specific patients., Competing Interests: The authors declare no conflict of interest.

Importance: In health care systems in which hospital reimbursement is based on a national mean length of stay (LOS), disadvantaged patients with an increased LOS may be a source of inefficiency. This implication has been reported in adult patients, but pediatric data have been scarce., Objective: To examine the association of patient socioeconomic status with hospital efficiency and financial balance in pediatrics., Design, Setting, and Participants: This cohort study obtained data from the French national hospital discharge database covering a 3-year period, from January 1, 2012, to December 31, 2014. Statistical analyses were performed between June 2016 and December 2018. All inpatient stays in hospital pediatric wards in mainland France by children older than 28 days or younger than 18 years (n = 4 121 187) were included. Admissions with coding errors or missing values for social disadvantage and/or cost calculations were excluded., Exposure: Social disadvantage was estimated with an ecological indicator, the FDep, available at the patient's postcode of residence and divided into national quintiles., Main Outcomes and Measures: Efficiency was assessed through the variations in patient LOS compared with different national mean LOS (for pediatric patients, pediatric patients with a similar condition, and pediatric patients with a similar condition and severity level). Hospital financial balance was assessed at the admission level through the ratio of production costs to revenues and at the hospital level through the difference between aggregated revenues and production costs. Multivariate regression models examined the association between these indicators and socioeconomic status., Results: A total of 4 121 187 admissions were included (2 336 540 [56.7%] male; mean [SD] age, 7.4 [5.8] years). In all, 1 561 219 patients (37.9%) were in the 2 most disadvantaged quintiles. Patient LOS was shorter than the national mean LOS (mean [SD], 1.73 [4.21] days) for patients in the least disadvantaged quintile and longer for those in the more disadvantaged quintile (mean [SD], 1.67 [4.33] days vs 1.82 [4.14] days). This difference was higher for diagnosis related groups that included both adult and pediatric patients (mean [SD], 1.46 [4.22] days vs 1.61 [4.13] days) compared with those dedicated to pediatric patients (2.22 [4.13] days vs 2.12 [4.53] days). Patients in the most disadvantaged quintile were associated with a 3.2% increase in LOS (odds ratio, 1.0322; 95% CI, 1.0302-1.0341) compared with the national mean LOS. Social disadvantage was also associated with a significant increase in financial deficit for hospitals with 20% to 60% of patients in the 2 most disadvantaged quintiles (estimate: -€146 389; 95% CI, -€279 566 to -€13 213)., Conclusions and Relevance: Patient socioeconomic status appears to be statistically significantly associated with an increase in LOS and cost in French hospitals with pediatric departments. This finding suggests that initiating reform in hospital payment methods may improve resource allocation efficiency and equity in access to pediatric care.

Aims: There is a need of more quantitative standardised data to compare local Mental Health Systems (MHSs) across international jurisdictions. Problems related to terminological variability and commensurability in the evaluation of services hamper like-with-like comparisons and hinder the development of work in this area. This study was aimed to provide standard assessment and comparison of MHS in selected local areas in Europe, contributing to a better understanding of MHS and related allocation of resources at local level and to lessen the scarcity in standard service comparison in Europe. This study is part of the Seventh Framework programme REFINEMENT (Research on Financing Systems' Effect on the Quality of Mental Health Care in Europe) project., Methods: A total of eight study areas from European countries with different systems of care (Austria, England, Finland, France, Italy, Norway, Romania, Spain) were analysed using a standard open-access classification system (Description and Evaluation of Services for Long Term Care in Europe, DESDE-LTC). All publicly funded services universally accessible to adults (≥18 years) with a psychiatric disorder were coded. Care availability, diversity and capacity were compared across these eight local MHS., Results: The comparison of MHS revealed more community-oriented delivery systems in the areas of England (Hampshire) and Southern European countries (Verona - Italy and Girona - Spain). Community-oriented systems with a higher proportion of hospital care were identified in Austria (Industrieviertel) and Scandinavian countries (Sør-Trøndelag in Norway and Helsinki-Uusimaa in Finland), while Loiret (France) was considered as a predominantly hospital-based system. The MHS in Suceava (Romania) was still in transition to community care., Conclusions: There is a significant variation in care availability and capacity across MHS of local areas in Europe. This information is relevant for understanding the process of implementation of community-oriented mental health care in local areas. Standard comparison of care provision in local areas is important for context analysis and policy planning.

Background: The FRANCE-2 registry (French Aortic National Corevalve and Edwards) previously reported good early- and medium-term clinical and echocardiographic efficacy for transcatheter aortic valve replacement. We here report 5-year follow-up results from the registry., Methods: The registry includes all consecutive patients undergoing transcatheter aortic valve replacement for severe aortic stenosis in France. Follow-up is scheduled at 30 days, 6 months, then annually from 1 to 5 years. Clinical events were defined according to the Valve Academic Research Consortium criteria, and hemodynamic structural valve deterioration (SVD) was defined according to the consensus statement by the European Association of Percutaneous Cardiovascular Interventions., Results: Between January 2010 and January 2012, 4201 patients were enrolled in 34 centers. Five-year vital status was available for 95.5% of patients; 88.1% had clinical evaluation or died. Overall, at 5 years, all-cause mortality was 60.8% (n=2478; 95% CI, 59.3% to 62.3%). The majority of cardiovascular events occurred in the first month after valve implantation, and incidence remained low thereafter, at <2% per year up to 5 years, except for heart failure. The rate of heart failure was 14.3% at 1 year, then decreased over time to <5% per year. In cumulative incidence function, the rates of severe SVD and moderate/severe SVD at 5 years were 2.5% and 13.3%, respectively. Mortality did not differ between patients with or without severe SVD (hazard ratio, 0.71; 95% CI, 0.47-1.07; P=0.1). Finally, in the population of patients with severe SVD, 1 patient (1.7%) experienced a stroke, and 8 patients presented ≥1 heart failure event (13.3%)., Conclusions: The 5-year follow-up results of the FRANCE-2 registry represent the largest long-term data set available in a high-risk population. In surviving patients, the low rate of clinical events and the low level of SVD after 1 year support the long-term efficacy of transcatheter aortic valve replacement in both types of transcatheter prosthesis featuring in the registry.

Purpose: Intensive care unit survivors suffer from prolonged impairment, reduced quality of life, and higher mortality rates after discharge compared to the general population. Socioeconomic status may play a partial but important role in mortality and recovery. Therefore, the detection of factors that are responsible for poor long-term outcomes would be beneficial in designing targeted interventions for at-risk populations., Methods: For an endpoint analysis, 1834 intensive care unit patients with known French Deprivation Index (FDep) scores were included from the French and euRopean Outcome reGistry in Intensive Care Units (FROG-ICU) study, which was a prospective, observational, multicenter cohort study performed in 20 French intensive care units in 13 different hospitals. Socioeconomic status was defined by using the FDep score [represented as quintiles when referring to the general French population, as quintiles when referring to the FROG-ICU cohort, or as dichotomized data (which was defined as a FDep ≤ 0 for nondeprived patients)] and by using a detailed social questionnaire that was completed 3 months after discharge. The primary outcome included an all-cause, 1-year mortality after ICU discharge when regarding socioeconomic status. The secondary outcomes included both ICU and hospital lengths of stay, both short- and medium-term mortality, and the quality of life, as assessed during the 1-year follow-up by using the Medical Outcome Survey Short Form-36 (SF-36). The Revised Impact of Event Scale (IES-R) was used to evaluate the symptoms of post-traumatic stress disorder, and the Hospital Anxiety and Depression Scale (HADS) was used to screen for anxiety and depression., Results: Of the 1447 patients who were discharged alive from the ICU, 19.2% died over the following year. No association was found between 1-year mortality and socioeconomic status, regardless of whether this association was analyzed in quintiles (p = 0.911 in the quintiles of the general French population; p = 0.589 in the quintiles of the FROG-ICU cohort itself) or as dichotomized data [nondeprived (n = 177; 1-year mortality of 18.2%) versus deprived (n = 97; 1-year mortality of 20.5%; p = 0.304)]. Moreover, no differences were found between the nondeprived and the deprived patients in the ICU and hospital lengths of stay, ICU mortalities, in-hospital mortalities, or 28-day mortalities. The SF-36 was below the score for the normal French population throughout the follow-up period. Socially deprived patients showed significantly lower median scores in the physical function subscale [55, interquartile range (IQR) (28.8-80) vs. 65, IQR (35-90); p = 0.014], the physical role subscale [25, IQR (0-75) vs. 33.3, IQR (0-100); p = 0.022], and the overall physical component scale [47.5, IQR (30-68.8) vs. 54.4, IQR (35-78.8); p = 0.010]. Up to 31.6% of survivors presented symptoms that indicated post-traumatic stress disorder, and up to 31.5% of survivors reported clinically meaningful symptoms of anxiety or depression., Conclusions: A lower socioeconomic status was associated with lower self-reported physical component scores in the nondeprived patients. Psychiatric symptoms are frequently reported after an ICU stay, and subsequent interventions should target those fields., Trial Registration: ClinicalTrials.gov NCT01367093; registered on June 6, 2011.

Aims: Although many mental health care systems provide care interventions that are not related to direct health care, little is known about the interfaces between the latter and core health care. 'Core health care' refers to services whose explicit aim is direct clinical treatment which is usually provided by health professionals, i.e., physicians, nurses, psychologists. 'Other care' is typically provided by other staff and includes accommodation, training, promotion of independence, employment support and social skills. In such a definition, 'other care' does not necessarily mean being funded or governed differently. The aims of the study were: (1) using a standard classification system (Description and Evaluation of Services and Directories in Europe for Long Term Care, DESDE-LTC) to identify 'core health' and 'other care' services provided to adults with mental health problems; and (2) to investigate the balance of care by analysing the types and characteristics of core health and other care services., Methods: The study was conducted in eight selected local areas in eight European countries with different mental health systems. All publicly funded mental health services, regardless of the funding agency, for people over 18 years old were identified and coded. The availability, capacity and the workforce of the local mental health services were described using their functional main activity or 'Main Types of Care' (MTC) as the standard for international comparison, following the DESDE-LTC system., Results: In these European study areas, 822 MTCs were identified as providing core health care and 448 provided other types of care. Even though one-third of mental health services in the selected study areas provided interventions that were coded as 'other care', significant variation was found in the typology and characteristics of these services across the eight study areas., Conclusions: The functional distinction between core health and other care overcomes the traditional division between 'health' and 'social' sectors based on governance and funding. The overall balance between core health and other care services varied significantly across the European sites. Mental health systems cannot be understood or planned without taking into account the availability and capacity of all services specifically available for this target population, including those outside the health sector.

Context: Previous studies, mainly originating from North America, suggest that women are less likely than men to obtain professorships in academic medical settings. However, research providing a comprehensive picture of such gender disparities in other national contexts and addressing associated contextual factors is lacking., Objectives: Our objectives were to assess gender differences in access to professorships in academic medical settings in France, to determine their evolution across regions and medical specialties and over time, and to identify the factors associated with the likelihood of a professor being a woman., Methods: We carried out a national administrative cohort study of all new professors appointed during 1989-2015 in all medical specialties in the whole of France. We first conducted a descriptive analysis of the percentage of professorships awarded to women and its variations by time, region and specialty. We then ran a logistic regression model to determine factors significantly associated with the likelihood of a professor being a woman., Results: Between 1989 and 2015, 3950 professors were appointed, of whom fewer than one in five were women. Female professors consistently represented a minority in all French regions and specialties over the study period. Although a small increase was observed over the years, women never represented more than 29% of newly appointed professors. After adjustments for other factors, the likelihood of a professor being a woman was significantly higher in specialties with a higher percentage of women among hospital practitioners, in regions with higher numbers of appointed professors and in recent years., Conclusions: Gender inequalities in career evolution exist in academic medical settings in France and have continued over time despite moderate improvements. Increased awareness based on scientific evidence is a first step towards reducing such inequalities., (© 2018 John Wiley & Sons Ltd and The Association for the Study of Medical Education.)

Background: Inpatient care accounts for the majority of mental health care costs and is not always beneficial. It can indeed have detrimental consequences if not used appropriately, and is unpopular among patients. As a consequence, its reduction is supported by international recommendations. Varying rates of psychiatric inpatient admissions therefore deserve to draw attention of researchers, clinicians and policy makers alike as such variations can challenge quality, equity and efficiency of care. In this context, our objectives were first to describe variations in psychiatric inpatient admission rates across the whole territory of mainland France, and second to identify their association with characteristics of the supply of care, which can be targeted by dedicated health policies., Methods: Our study was carried out in French psychiatric sectors' catchment areas for the year 2012. Inpatient admission rates per 100,000 adult inhabitants were calculated using data from the national psychiatric discharge database. Their variations were described numerically and graphically. We then carried out a negative binomial regression to identify characteristics of the supply of care (public and private care, health and social care, hospital and community-based care, specialised and non-specialised care) which were associated with these variations while adjusting our analysis for other relevant factors, in particular epidemiological differences., Results: Considerable variations in inpatient admission rates were observed between psychiatric sectors' catchment areas and were widespread on the French territory. Institutional characteristics of the hospital to which each sector was linked (private non-profit status, specialisation in psychiatry and participation to teaching activities and to emergency care) were associated with inpatient admission rates. Similarly, an increase in the availability of community-based private psychiatrists was associated with a decrease in the inpatient admission rate while an increase in the capacity of housing institutions for disabled individuals was associated with an increase in this rate., Conclusions: Our results advocate for a homogenous repartition of health and social care for mental disorders in lines with the health needs of the population served. This should apply particularly to community-based private psychiatrists, whose heterogeneity of repartition has often been underscored.

Background: Transcatheter aortic valve replacement (TAVR) has emerged as an alternative to surgical aortic valve replacement (SAVR), but unbiased data regarding evolution of the treatment of patients with aortic stenosis at the nationwide level are scarce., Objectives: This study sought to evaluate the number of aortic valve replacements (AVRs) performed in France, changes over time, and the effect of the adoption of TAVR., Methods: Based on a French administrative hospital-discharge database, the study collected all consecutive AVRs performed in France between 2007 and 2015., Results: A total of 131,251 interventions were performed: 109,317 (83%) SAVR and 21,934 (17%) TAVR. AVR linearly increased (from 10,892 to 18,704; p for trend <0.0001) mainly due to a marked increase in TAVR (from 244 to 6,722; p for trend = 0.0004), whereas SAVR remained stable (from 10,892 to 11,982; p for trend = 0.18). Parallel to a decrease in the Charlson index (p for trend <0.05), SAVR and TAVR in-hospital mortality rates significantly declined (both p for trend <0.01). The number of TAVRs significantly increased in all age categories (<75, 75 to 79, 80 to 84, and ≥85 years of age; all p for trend = 0.003), but reached or even exceeded SAVR in the 2 oldest categories. Although mortality rates declined for both isolated SAVR and TAVR, it became similar or slightly lower for TAVR than for isolated SAVR in 2015 in the 3 oldest age categories even if it did not reach statistical significance (p = 0.66, p = 0.47, and p = 0.06, respectively)., Conclusions: The number of AVRs markedly increased in France between 2007 and 2015 due to the wide adoption of TAVR, which represented one-third of all AVRs in 2015. Patients' profile improved, suggesting that patients are referred earlier, and in-hospital mortality declined in all AVR subsets. Despite a worse clinical profile, the immediate outcome of TAVR compared favorably to isolated SAVR in patients >75 years of age. The results may have major implications for clinical practice and policymakers., (Copyright © 2018 American College of Cardiology Foundation. Published by Elsevier Inc. All rights reserved.)

Background: Involuntary psychiatric care remains controversial. Geographic disparities in its use can challenge the appropriateness of the care provided when they do not result from different health needs of the population. These disparities should be reduced through dedicated health policies. However, their association with the supply of health and social care, which could be targeted by such policies, has been insufficiently studied. Our objectives were therefore to describe geographic variations in involuntary admission rates across France and to identify the characteristics of the supply of care which were associated with these variations., Methods: Involuntary admission rate per 100,000 adult inhabitants was calculated in French psychiatric sectors' catchment areas using 2012 data from the national psychiatric discharge database. Its variations were first described numerically and graphically. Several factors potentially associated with these variations were then considered in a negative binomial regression with an offset term accounting for the size of catchment areas. They included characteristics of the supply of care (public and private care, health and social care, hospital and community-based care, specialised and non-specialised care) as well as adjustment factors related to epidemiological characteristics of the population of each sector's catchment area and its level of urbanization. Such variables were extracted from complementary administrative databases. Supply characteristics associated with geographic variations were identified using a significance level of 0.05., Results: Significant variations in involuntary admission rates were observed between psychiatric sectors' catchment areas with a coefficient of variation close to 80%. These variations were associated with some characteristics of the supply of health and social care in the sectors' catchment areas. Notably, an increase in the availability of community-based private psychiatrists and the capacity of housing institutions for disabled individuals was associated with a decrease in involuntary admission rates while an increase in the availability of general practitioners was associated with an increase in those rates., Conclusions: There is evidence of considerable variations in involuntary admission rates between psychiatric sectors' catchment areas. Our results provide lines of thoughts to reduce such variations, in particular by supporting an increase in the availability of upstream and downstream care in the community.

Comparing mental health systems across countries is difficult because of the lack of an agreed upon terminology covering services and related financing issues. Within the European Union project REFINEMENT, international mental health care experts applied an innovative mixed "top-down" and "bottom-up" approach following a multistep design thinking strategy to compile a glossary on mental health systems, using local services as pilots. The final REFINEMENT glossary consisted of 432 terms related to service provision, service utilisation, quality of care and financing. The aim of this study was to describe the iterative process and methodology of developing this glossary.

Background: Inpatient psychiatric readmissions are often used as an indicator of the quality of care and their reduction is in line with international recommendations for mental health care. Research on variations in inpatient readmission rates among mental health care providers is therefore of key importance as these variations can impact equity, quality and efficiency of care when they do not result from differences in patients' needs., Aims of the Study: Our objectives were first to describe variations in inpatient readmission rates between public mental health care providers in France on a nationwide scale, and second, to identify their association with patient, health care providers and environment characteristics., Methods: We carried out a study for the year 2012 using data from ten administrative national databases. 30-day readmissions in inpatient care were identified in the French national psychiatric discharge database. Variations were described numerically and graphically between French psychiatric sectors and factors associated with these variations were identified by carrying out a multi-level logistic regression accounting for the hierarchical structure of the data., Results: Significant practice variations in 30-day inpatient readmission rates were observed with a coefficient of variation above 50%. While a majority of those variations was related to differences within sectors, individual patient characteristics explained a lower part of the variations resulting from differences between sectors than the characteristics of sectors and of their environment. In particular, an increase in the mortality rate and in the acute admission rate for somatic disorders in sectors' catchment area was associated with a decrease in the probability of 30-day readmission. Similarly, an increase in the number of psychiatric inpatient beds in private for-profit hospitals per 1,000 inhabitants in sectors' catchment area was associated with a decrease in this probability, which also varied with overall sectors' case-mix characteristics and with the level of urbanisation of the area., Discussion: The extent of the variations and the factors associated with it question the adequacy of care and suggest that some of them may be unwarranted. Our findings should however be interpreted in consideration of several limits inherent to data quality and availability as we relied on information from administrative databases. While we considered a wide range of factors potentially associated with variations in 30-day readmissions, our model indeed only explained a limited part of the variations resulting from differences between sectors., Implications for Health Policies: Our findings underscored that practice variations in psychiatry are a reality that merits the full attention of decision makers as they can impact the quality, equity and efficiency of care. A specific data system should be established to monitor practice variations in routine to promote transparency and accountability., Implications for Further Research: Few associations were found between variations in 30-day inpatient readmissions and the supply of care. The routine collection of detailed organizational characteristics of health care providers at a national level should be supported to facilitate additional research work, both in France and in other contexts.

Background: Intensive care unit (ICU) survivors have reduced long-term survival compared to the general population. Identifying parameters at ICU discharge that are associated with poor long-term outcomes may prove useful in targeting an at-risk population. The main objective of the study was to identify clinical and biological determinants of death in the year following ICU discharge., Methods: FROG-ICU was a prospective, observational, multicenter cohort study of ICU survivors followed 1 year after discharge, including 21 medical, surgical or mixed ICUs in France and Belgium. All consecutive patients admitted to intensive care with a requirement for invasive mechanical ventilation and/or vasoactive drug support for more than 24 h following ICU admission and discharged from ICU were included. The main outcome measure was all-cause mortality at 1 year after ICU discharge. Clinical and biological parameters on ICU discharge were measured, including the circulating cardiovascular biomarkers N-terminal pro-B type natriuretic peptide, high-sensitive troponin I, bioactive-adrenomedullin and soluble-ST2. Socioeconomic status was assessed using a validated deprivation index (FDep)., Results: Of 1570 patients discharged alive from the ICU, 333 (21%) died over the following year. Multivariable analysis identified age, comorbidity, red blood cell transfusion, ICU length of stay and abnormalities in common clinical factors at the time of ICU discharge (low systolic blood pressure, temperature, total protein, platelet and white cell count) as independent factors associated with 1-year mortality. Elevated biomarkers of cardiac and vascular failure independently associated with 1-year death when they are added to multivariable model, with an almost 3-fold increase in the risk of death when combined (adjusted odds ratio 2.84 (95% confidence interval 1.73-4.65), p < 0.001)., Conclusions: The FROG-ICU study identified, at the time of ICU discharge, potentially actionable clinical and biological factors associated with poor long-term outcome after ICU discharge. Those factors may guide discharge planning and directed interventions., Trial Registration: ClinicalTrials.gov NCT01367093 . Registered on 6 June 2011.

Introduction: There is a lack of data on health care consumption of patients suffering from schizophrenia, as well as on the related health care costs. Factors associated with health care costs have not been widely studied, whereas knowledge on this topic would allow identifying risk factors and delineating strategies to improve patients' health and follow-up, likely to also decrease health care costs. The aim of this study was to estimate the average direct health care cost of patients with schizophrenia in France and to identify the factors associated with this cost., Methods: The study population included patients with schizophrenia enrolled in the FondaMental Advanced Centers of Expertise for Schizophrenia cohort. We accounted for the costs directly related to the treatment of schizophrenia. They included the costs of hospitalizations (full- and part-time), psychiatric ambulatory consultations and medications. We studied three categories of factors potentially associated with direct health care costs: demographic, socioeconomic and clinical characteristics., Results: Three hundred and ninety five patients with schizophrenia were included. The mean (median) annual direct health care cost per patient amounted to €14,995 (€3,435). A lower level of functioning and being single were associated with a higher cost. A significant association between the expert center of inclusion and the direct health care cost of schizophrenia was also highlighted., Conclusion: Our results highlighted the significant cost of schizophrenia and suggest that improvement in patient care, based on well-validated targeted therapeutic interventions such as psycho-education and cognitive rehabilitation, could reduce worsening in symptom severity and therefore decrease health care costs., (Copyright © 2017 The Authors. Published by Elsevier B.V. All rights reserved.)

Background: Coronary artery disease (CAD) is common in patients undergoing transcatheter aortic valve replacement (TAVR). However, the impact of CAD distribution before TAVR on short- and long-term prognosis remains unclear., Hypothesis: We hypothesized that the long-term clinical impact differs according to CAD distribution in patients undergoing TAVR using the FRench Aortic National CoreValve and Edwards (FRANCE-2) registry., Methods: FRANCE-2 is a national French registry including all consecutive TAVR performed between 2010 and 2012 in 34 centers. Three-year mortality was assessed in relation to CAD status. CAD was defined as at least 1 coronary stenosis >50%., Results: A total of 4201 patients were enrolled in the registry. For the present analysis, we excluded patients with a history of coronary artery bypass. CAD was reported in 1252 patients (30%). Half of the patients presented with coronary multivessel disease. CAD extent was associated with an increase in cardiovascular risk profile and in logistic EuroSCORE (European System for Cardiac Operative Risk Evaluation) (from 19.3% ± 12.8% to 21.9% ± 13.5%, P < 0.001). Mortality at 30 days and 3 years was 9% and 44%, respectively, in the overall population. In multivariate analyses, neither the presence nor the extent of CAD was associated with mortality at 3 years (presence of CAD, hazard ratio [HR]: 0.90; 95% confidence interval [CI]: 0.78-1.07). A significant lesion of the left anterior descending (LAD) was associated with higher 3-year mortality (HR: 1.42; 95% CI: 1.10-1.87)., Conclusions: CAD is not associated with decreased short- and long-term survival in patients undergoing TAVR. The potential deleterious effect of LAD disease on long-term survival and the need for revascularization before or at the time of TAVR should be validated in a randomized control trial., (© 2017 Wiley Periodicals, Inc.)

Purpose: Health-related quality of life (HRQoL) is a widely used concept in the assessment of health care. Some generic HRQoL instruments, based on specific algorithms, can generate utility scores which reflect the preferences of the general population for the different health states described by the instrument. This study aimed to investigate the relationships between utility scores and potentially associated factors in patients with mental disorders followed in inpatient and/or outpatient care settings using two statistical methods., Methods: Patients were recruited in four psychiatric sectors in France. Patient responses to the SF-36 generic HRQoL instrument were used to calculate SF-6D utility scores. The relationships between utility scores and patient socio-demographic, clinical characteristics, and mental health care utilization, considered as potentially associated factors, were studied using OLS and quantile regressions., Results: One hundred and seventy six patients were included. Women, severely ill patients and those hospitalized full-time tended to report lower utility scores, whereas psychotic disorders (as opposed to mood disorders) and part-time care were associated with higher scores. The quantile regression highlighted that the size of the associations between the utility scores and some patient characteristics varied along with the utility score distribution, and provided more accurate estimated values than OLS regression., Conclusions: The quantile regression may constitute a relevant complement for the analysis of factors associated with utility scores. For policy decision-making, the association of full-time hospitalization with lower utility scores while part-time care was associated with higher scores supports the further development of alternatives to full-time hospitalizations.

Background: Some patients who are at high surgical risk because of multiple co-morbidities undergo balloon aortic valvuloplasty (BAV) as a bridge therapy towards transcatheter aortic-valve implantation (TAVI)., Aim: The aim of this study was to compare the clinical course of patients with or without previous BAV who underwent TAVI and were included in the FRANCE 2 registry., Methods: From January 2010 to December 2011, 3953 patients underwent TAVI. Survival analysis was done by multivariable regression and propensity-score analysis to adjust for confounders., Results: Patients in the previous BAV group (n=664, 16.8%) were older than patients in the primary TAVI group. The logistic EuroSCORE I and the rates of co-morbidities and symptoms were higher in the previous BAV group. Procedural success was similar in both groups, as was postprocedural aortic regurgitation grade≥2/4. The 1-month mortality rates from all causes were 12.5 and 8.7%, respectively, in the previous BAV and primary TAVI groups (P=0.001). The 1-month to 1-year mortality rates were similar in both groups. Previous BAV was not an independent predictor of 1-month mortality (hazard ratio 1.44, 95% confidence interval 0.90-2.34; P=0.14) or 1-month to 1-year mortality., Conclusions: Crude 1-month mortality was higher in patients with previous BAV. Nevertheless, precarious preoperative status, but not previous BAV, was associated with mortality, and is the only marker that should be considered as detrimental at the time of preTAVI reassessment., (Copyright © 2017 Elsevier Masson SAS. All rights reserved.)

Objective: The development of alternatives to full-time hospitalization in psychiatry is limited because consensus about the benefits of such alternatives is lacking. This study assessed whether the development of such alternatives in French psychiatric sectors was associated with a reduction in involuntary inpatient care, taking into account other factors that are potentially associated with involuntary admission., Methods: Data on whether a patient had at least one involuntary full-time admission in 2012 were extracted from the French national discharge database for psychiatric care. The development of alternatives to full-time hospitalization was estimated as the percentage of human resources allocated to these alternatives out of all human resources allocated to psychiatry, measured at the level of the hospital hosting each sector. Other factors potentially associated with involuntary admission (characteristics of patients, health care providers, and the environment) were extracted from administrative databases, and a multilevel logistic model was carried out to account for the nested structure of the data., Results: Significant variations were observed between psychiatric sectors in rates of involuntary inpatient admissions. A large portion of the variation was explained by characteristics of the sectors. A significant negative association was found between involuntary admissions and the development of alternatives to full-time hospitalization, after adjustment for other factors associated with involuntary admissions., Conclusions: Findings suggest that the development of alternatives to full-time hospitalization is beneficial for quality of care, given that it is negatively associated with involuntary full-time admissions. The reduction of such admissions aligns with international recommendations for psychiatric care.

Background: An increasing number of model-based studies that evaluate the cost effectiveness of treatments for depression are being published. These studies have different characteristics and use different simulation methods., Objective: We aimed to systematically review model-based studies evaluating the cost effectiveness of treatments for depression and examine which modelling technique is most appropriate for simulating the natural course of depression., Methods: The literature search was conducted in the databases PubMed, EMBASE and PsycInfo between 1 January 2002 and 1 October 2016. Studies were eligible if they used a health economic model with quality-adjusted life-years or disability-adjusted life-years as an outcome measure. Data related to various methodological characteristics were extracted from the included studies. The available modelling techniques were evaluated based on 11 predefined criteria., Results: This methodological review included 41 model-based studies, of which 21 used decision trees (DTs), 15 used cohort-based state-transition Markov models (CMMs), two used individual-based state-transition models (ISMs), and three used discrete-event simulation (DES) models. Just over half of the studies (54%) evaluated antidepressants compared with a control condition. The data sources, time horizons, cycle lengths, perspectives adopted and number of health states/events all varied widely between the included studies. DTs scored positively in four of the 11 criteria, CMMs in five, ISMs in six, and DES models in seven., Conclusion: There were substantial methodological differences between the studies. Since the individual history of each patient is important for the prognosis of depression, DES and ISM simulation methods may be more appropriate than the others for a pragmatic representation of the course of depression. However, direct comparisons between the available modelling techniques are necessary to yield firm conclusions.

Hepatocellular carcinoma (HCC) is the leading cause of death in patients with cirrhosis. Patients outside clinical trials seldom benefit from evidence-based monitoring. The objective of this study was to estimate the cost-effectiveness of complying with HCC screening guidelines. The economic evaluation compared surveillance of patients with cirrhosis as recommended by the guidelines ("gold-standard monitoring") to "real-life monitoring" from the health care system perspective. A Markov model described the history of the disease and treatment course including current first-line curative treatment: liver resection, radiofrequency ablation (RFA), and liver transplantation. Transition probabilities were derived mainly from two French cohorts, CIRVIR and CHANGH. Costs were computed using French and U.S. tariffs. Effectiveness was measured in life years gained (LYG). An incremental cost-effectiveness ratio (ICER) was calculated for a 10-year horizon and tested with one-way and probabilistic sensitivity analyses. The cost difference between the two groups was $648 ($87,476 in the gold-standard monitoring group vs. $86,829 in the real-life monitoring group) in France and $11,965 ($93,795 vs. $81,829) in the United States. Survival increased by 0.37 years (7.18 vs. 6.81 years). The ICER was $1,754 per LYG in France and $32,415 per LYG in the United States. The health gain resulted from earlier diagnosis and access to first-line curative treatments, among which RFA provided the best value for money., Conclusion: Our results indicate that gold-standard monitoring for patients with cirrhosis is cost-effective, attributed to a higher probability of benefiting from a curative treatment and so a higher survival probability. (Hepatology 2017;65:1237-1248)., (© 2017 by the American Association for the Study of Liver Diseases.)

International recommendations for mental health care have advocated for a reduction in the length of stay (LOS) in full-time hospitalization and the development of alternatives to full-time hospitalizations (AFTH) could facilitate alignment with those recommendations. Our objective was therefore to assess whether the development of AFTH in French psychiatric sectors was associated with a reduction in the LOS in full-time hospitalization. Using data from the French national discharge database of psychiatric care, we computed the LOS of patients admitted for full-time hospitalization. The level of development of AFTH was estimated by the share of human resources allocated to those alternatives in the hospital enrolling the staff of each sector. Multi-level modelling was carried out to adjust the analysis on other factors potentially associated with the LOS (patients', psychiatric sectors' and environmental characteristics). We observed considerable variations in the LOS between sectors. Although the majority of these variations resulted from patients' characteristics, a significant negative association was found between the LOS and the development of AFTH, after adjusting for other factors. Our results provide first evidence of the impact of the development of AFTH on mental health care and will provide a lever for policy makers to further develop these alternatives.

Background: Health-economic models are used to evaluate the long-term cost-effectiveness of an intervention and typically include treatment as usual (TAU) as comparator. Part of the data used for these models are acquired from the literature and thus valid information is needed on the effects of TAU on depression. The aim of the current meta-analysis was to examine positive and negative outcomes of major depression for patients receiving TAU., Methods: We conducted a systematic literature search in PubMed, EMBASE, PsycInfo, and the Cochrane Central Register of Controlled Trials. Eligible studies were randomized controlled trials including a TAU group for depression. The quality of the included studies was assessed using the criteria described in the "Risk of bias assessment tool". Four separate meta-analyses were performed to estimate remission, response, reliable change and deterioration rates at short-term (≤6 months from baseline)., Results: Thirty-eight studies including 2099 patients in the TAU were identified. Nine studies (24%) met five or six quality criteria, 17 studies (44%) met three or four quality criteria and 12 studies (32%) met one or two quality criteria. After adjusting for publication bias, the first meta-analysis (n=33) showed that 33% of the patients remitted from depression. The second meta-analysis (n=13) demonstrated that 27% of the patients responded to treatment, meaning that their depressive symptom decreased at least 50% from baseline to follow-up measurement. The third meta-analysis (n=7) indicated that 31% of the patients showed a reliable change, meaning that their depressive symptoms improved more than expected by random variation alone. Finally, 12% of the patients deteriorated, meaning that their depressive symptoms became more severe., Limitations: Statistical heterogeneity was substantial in most analyses and was not fully explained by subgroup analyses. The quality of the included studies was moderate. This may result in overestimation of the true effects., Conclusions: The treatments labelled as TAU for depression were clinically and statistically heterogeneous. We demonstrated that a few patients benefited from TAU and a small number of patients suffered from worsened depressive symptoms at the short term. The results can be included in health-economic models that compare depression treatments to TAU., (Copyright © 2016 Elsevier B.V. All rights reserved.)

Background: The integration of digital treatments into national mental health services is on the agenda in the European Union. The E-COMPARED consortium conducted a survey aimed at exploring stakeholders' knowledge, acceptance and expectations of digital treatments for depression, and at identifying factors that might influence their opinions when considering the implementation of these approaches., Method: An online survey was conducted in eight European countries: France, Germany, Netherlands, Poland, Spain, Sweden, Switzerland and The United Kingdom. Organisations representing government bodies, care providers, service-users, funding/insurance bodies, technical developers and researchers were invited to participate in the survey. The participating countries and organisations reflect the diversity in health care infrastructures and e-health implementation across Europe., Results: A total of 764 organisations were invited to the survey during the period March-June 2014, with 175 of these organisations participating in our survey. The participating stakeholders reported moderate knowledge of digital treatments and considered cost-effectiveness to be the primary incentive for integration into care services. Low feasibility of delivery within existing care services was considered to be a primary barrier. Digital treatments were regarded more suitable for milder forms of depression. Stakeholders showed greater acceptability towards blended treatment (the integration of face-to-face and internet sessions within the same treatment protocol) compared to standalone internet treatments. Organisations in countries with developed e-health solutions reported greater knowledge and acceptability of digital treatments., Conclusion: Mental health stakeholders in Europe are aware of the potential benefits of digital interventions. However, there are variations between countries and stakeholders in terms of level of knowledge about such interventions and their feasibility within routine care services. The high acceptance of blended treatments is an interesting finding that indicates a gradual integration of technology into clinical practice may fit the attitudes and needs of stakeholders. The potential of the blended treatment approach, in terms of enhancing acceptance of digital treatment while retaining the benefit of cost-effectiveness in delivery, should be further explored., Funding: The E-COMPARED project has received funding from the European Union Seventh Framework Programme (FP7/2007-2013) under grant agreement no. 603098.

Background: Transcatheter aortic valve replacement (TAVR) has revolutionized management of high-risk patients with severe aortic stenosis. However, survival and the incidence of severe complications have been assessed in relatively small populations and/or with limited follow-up., Objectives: This report details late clinical outcome and its determinants in the FRANCE-2 (FRench Aortic National CoreValve and Edwards) registry., Methods: The FRANCE-2 registry prospectively included all TAVRs performed in France. Follow-up was scheduled at 30 days, at 6 months, and annually from 1 to 5 years. Standardized VARC (Valve Academic Research Consortium) outcome definitions were used., Results: A total of 4,201 patients were enrolled between January 2010 and January 2012 in 34 centers. Approaches were transarterial (transfemoral 73%, transapical 18%, subclavian 6%, and transaortic or transcarotid 3%) or, in 18% of patients, transapical. Median follow-up was 3.8 years. Vital status was available for 97.2% of patients at 3 years. The 3-year all-cause mortality was 42.0% and cardiovascular mortality was 17.5%. In a multivariate model, predictors of 3-year all-cause mortality were male sex (p < 0.001), low body mass index, (p < 0.001), atrial fibrillation (p < 0.001), dialysis (p < 0.001), New York Heart Association functional class III or IV (p < 0.001), higher logistic EuroSCORE (p < 0.001), transapical or subclavian approach (p < 0.001 for both vs. transfemoral approach), need for permanent pacemaker implantation (p = 0.02), and post-implant periprosthetic aortic regurgitation grade ≥2 of 4 (p < 0.001). Severe events according to VARC criteria occurred mainly during the first month and subsequently in <2% of patients/year. Mean gradient, valve area, and residual aortic regurgitation were stable during follow-up., Conclusions: The FRANCE-2 registry represents the largest database available on late results of TAVR. Late mortality is largely related to noncardiac causes. Incidence rates of severe events are low after the first month. Valve performance remains stable over time., (Copyright © 2016 American College of Cardiology Foundation. Published by Elsevier Inc. All rights reserved.)

Background: Effective, accessible, and affordable depression treatment is of high importance considering the large personal and economic burden of depression. Internet-based treatment is considered a promising clinical and cost-effective alternative to current routine depression treatment strategies such as face-to-face psychotherapy. However, it is not clear whether research findings translate to routine clinical practice such as primary or specialized mental health care. The E-COMPARED project aims to gain knowledge on the clinical and cost-effectiveness of blended depression treatment compared to treatment-as-usual in routine care., Methods/design: E-COMPARED will employ a pragmatic, multinational, randomized controlled, non-inferiority trial in eight European countries. Adults diagnosed with major depressive disorder (MDD) will be recruited in primary care (Germany, Poland, Spain, Sweden, and the United Kingdom) or specialized mental health care (France, The Netherlands, and Switzerland). Regular care for depression is compared to "blended" service delivery combining mobile and Internet technologies with face-to-face treatment in one treatment protocol. Participants will be followed up at 3, 6, and 12 months after baseline to determine clinical improvements in symptoms of depression (primary outcome: Patient Health Questionnaire-9), remission of depression, and cost-effectiveness. Main analyses will be conducted on the pooled data from the eight countries (n = 1200 in total, 150 participants in each country)., Discussion: The E-COMPARED project will provide mental health care stakeholders with evidence-based information and recommendations on the clinical and cost-effectiveness of blended depression treatment., Trial Registration: France: ClinicalTrials.gov NCT02542891 . Registered on 4 September 2015; Germany: German Clinical Trials Register DRKS00006866 . Registered on 2 December 2014; The Netherlands: Netherlands Trials Register NTR4962 . Registered on 5 January 2015; Poland: ClinicalTrials.Gov NCT02389660 . Registered on 18 February 2015; Spain: ClinicalTrials.gov NCT02361684 . Registered on 8 January 2015; Sweden: ClinicalTrials.gov NCT02449447 . Registered on 30 March 2015; Switzerland: ClinicalTrials.gov NCT02410616 . Registered on 2 April 2015; United Kingdom: ISRCTN registry, ISRCTN12388725 . Registered on 20 March 2015.

Objective: The aim of this study was to determine the economic burden from a societal perspective and the health-related quality of life (HRQOL) of patients with histiocytosis in Europe., Methods: We conducted a cross-sectional study of patients with histiocytosis from France, Germany, Italy, Spain, Bulgaria, the UK, and Sweden. Data on demographic characteristics, health resource utilisation, informal care, loss of labour productivity and HRQOL were collected from the questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) questionnaire., Results: A total of 134 patients (35 France, 32 Germany, 30 Italy, 24 Spain, 7 Bulgaria, 4 UK and 2 Sweden) completed the questionnaire. The average annual costs ranged from € 6832 to € 33,283 between countries, the year of reference being 2012. Estimated direct healthcare costs ranged from € 1698 to € 18,213; direct nonhealthcare costs ranged from € 2936 to € 17,622 and labour productivity losses ranged from € 1 to € 8855. The mean EQ-5D score for adult histiocytosis patients was estimated at between 0.32 and 0.85, and the mean EQ-5D visual analogue scale score was estimated at between 50.00 and 66.50., Conclusion: The main strengths of this study lie in our bottom-up approach to costing and in the evaluation of histiocytosis patients from a broad perspective (societal costs). This type of analysis is very scarce in international literature for rare diseases in comparison with other illnesses. We conclude that histiocytosis patients incur considerable societal costs and experience substantial deterioration in HRQOL.

Objective: The aim of this study was to determine the economic burden from a societal perspective and the health-related quality of life (HRQOL) of patients with juvenile idiopathic arthritis (JIA) in Europe., Methods: We conducted a cross-sectional study of patients with JIA from Germany, Italy, Spain, France, the United Kingdom, Bulgaria, and Sweden. Data on demographic characteristics, healthcare resource utilization, informal care, labor productivity losses, and HRQOL were collected from the questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D-5L) questionnaire., Results: A total of 162 patients (67 Germany, 34 Sweden, 33 Italy, 23 United Kingdom, 4 France, and 1 Bulgaria) completed the questionnaire. Excluding Bulgarian results, due to small sample size, country-specific annual health care costs ranged from €18,913 to €36,396 (reference year: 2012). Estimated direct healthcare costs ranged from €11,068 to €22,138; direct non-healthcare costs ranged from €7837 to €14,155 and labor productivity losses ranged from €0 to €8715. Costs are also shown to differ between children and adults. The mean EQ-5D index score for JIA patients was estimated at between 0.44 and 0.88, and the mean EQ-5D visual analogue scale score was estimated at between 62 and 79., Conclusions: JIA patients incur considerable societal costs and experience substantial deterioration in HRQOL in some countries. Compared with previous studies, our results show a remarkable increase in annual healthcare costs for JIA patients. Reasons for the increase are the inclusion of non-professional caregiver costs, a wider use of biologics, and longer hospital stays.

Objective: To estimate the social/economic costs of fragile X syndrome (FXS) in Europe and to assess the health-related quality of life (HRQOL) of patients and caregivers., Methods: A cross-sectional study was conducted in a sample of European countries. Patients were recruited through patients' associations. Data on their resource use and absence from the labour market were retrospectively obtained from an online questionnaire. Costs were estimated by a bottom-up approach and the EuroQol-5 Domain (EQ-5D) questionnaire was used to measure patients' and caregivers' HRQOL., Results: Five countries were included in the analysis. The mean annual cost of FXS per patient varied from €4951 in Hungary to €58,862 in Sweden. Direct non-healthcare costs represented the majority of costs in all countries but there were differences in the share incurred by formal and informal care among those costs. Costs were also shown to differ between children and adults. Mean EQ-5D utility score for adult patients varied from 0.52 in France (n = 42) to 0.73 in Hungary (n = 2), while for caregivers this score was consistently inferior to 0.87., Conclusion: Our findings underline that, although its prevalence is low, FXS is costly from a societal perspective. They support the development of tailored policies to reduce the consequences of FXS on both patients and their relatives.

Background: The aim of this study was to determine the social/economic costs and health-related quality of life (HRQOL) of patients with epidermolysis bullosa (EB) in eight EU member states., Methods: We conducted a cross-sectional study of patients with EB from Bulgaria, France, Germany, Hungary, Italy, Spain, Sweden and the United Kingdom. Data on demographic characteristics, health resource utilisation, informal care, labour productivity losses, and HRQOL were collected from the questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) questionnaire., Results: A total of 204 patients completed the questionnaire. Average annual costs varied from country to country, and ranged from €9509 to €49,233 (reference year 2012). Estimated direct healthcare costs ranged from €419 to €10,688; direct non-healthcare costs ranged from €7449 to €37,451 and labour productivity losses ranged from €0 to €7259. The average annual cost per patient across all countries was estimated at €31,390, out of which €5646 accounted for direct health costs (18.0 %), €23,483 accounted for direct non-healthcare costs (74.8 %), and €2261 accounted for indirect costs (7.2 %). Costs were shown to vary across patients with different disability but also between children and adults. The mean EQ-5D score for adult EB patients was estimated at between 0.49 and 0.71 and the mean EQ-5D visual analogue scale score was estimated at between 62 and 77., Conclusion: In addition to its negative impact on patient HRQOL, our study indicates the substantial social/economic burden of EB in Europe, attributable mostly to high direct non-healthcare costs.

Objective: The aim of this study was to determine the economic burden from a societal perspective and the health-related quality of life (HRQOL) of patients with Duchenne muscular dystrophy (DMD) in Europe., Methods: We conducted a cross-sectional study of patients with DMD from Bulgaria, France, Germany, Hungary, Italy, Spain, Sweden, and the UK. Data on demographic characteristics, healthcare resource utilization, informal care, labor productivity losses, and HRQOL were collected from the questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) questionnaire. Costs have been estimated from a societal perspective adopting a bottom-up approach., Results: A total of 422 questionnaires were included in the study; 268 of which were collected from patients with DMD and 154 from caregivers. The average annual cost per person in 2012 ranged from €7657 in Hungary to €58,704 in France. Direct non-healthcare costs are the main component of whole costs and informal care is the main driver of non-healthcare costs. Costs are also shown to differ between children and adults. With regard to HRQOL of adult patients, the EQ-5D VAS score and EQ-5D index scores were 50.5 and 0.24, respectively. The corresponding EQ-5D VAS and EQ-5D index scores for caregivers were 74.7 and 0.71, respectively., Conclusions: We have estimated the average annual cost per patient with DMD in eight European countries adopting a social perspective, and to our knowledge this is the first study with such a wide perspective. The results on costs show a considerable gap between Eastern and Western European countries. Non-healthcare costs range from 64 to 89 % of overall costs and informal care is to a great extent the main driver of this cost category. The HRQOL of people with DMD is much lower than that of the general population.

Objectives: Our goal was to provide data on the economic burden and health-related quality of life (HRQOL) of patients with cystic fibrosis (CF) and their caregivers in Europe., Methods: A cross-sectional study was carried out on adults and children with CF in eight European countries. Patients completed an anonymous questionnaire regarding their socio-demographic characteristics, use of healthcare services and presence of a caregiver. Costs were calculated with a bottom-up approach using unit costs from each participating country, and HRQOL was assessed using EQ-5D. The principal caregiver also answered a questionnaire on their characteristics, HRQOL and burden., Results: A total of 905 patients with CF was included (399 adults and 506 children). The total average annual cost per patient varied from €21,144 in Bulgaria to €53,256 in Germany. Adults had higher direct healthcare costs than children, but children had much higher informal care costs (P < 0.0001). Total costs increased with patients' level of dependence. In adults, mean utility fell between 0.640 and 0.870, and the visual analogue scale ranged from 46.0 to 69.7. There was no difference in caregiver HRQOL regardless of whether they cared for an adult or a child. However, caregivers who looked after a child had a significantly higher burden (P = 0.0013)., Conclusions: Our study highlights the burden of CF in terms of costs and decreased HRQOL for both patients and their caregivers throughout Europe.

Objectives: To assess the health-related quality of life (HRQOL) of patients with mucopolysaccharidosis (MPS) and their caregivers and to quantify the disease-related costs from a societal perspective., Methods: In the context of a multi-country study of rare diseases (BURQOL-RD project), a cross-sectional survey was performed among MPS patients in seven European countries. Data on demographic characteristics, health resource utilization, informal care, and loss of labor productivity were collected. The EQ-5D, Barthel index (BI), and Zarit burden interview (ZBI) questionnaires were used to assess patients' and their informal caregivers' quality of life, patients' functional ability, and caregivers' burden, respectively., Results: Altogether, 120 patients (children 62 %, females 40 %) and 66 caregivers completed the questionnaire. Patients' mean age was 16.5 years and median age at diagnosis was 3 years. Adult patients' average EQ-5D and EQ VAS scores varied across countries from 0.13 to 0.43 and 30.0 to 62.2, respectively, mean BI was 46.7, and ZBI was 32.7. Mean informal care time was 51.3 h/week. The mean total annual cost per patient (reference year 2012) was €24,520 in Hungary, €25,993 in France, €84,921 in Italy, €94,384 in Spain, and €209,420 in Germany. Costs are also shown to differ between children and adults. Direct costs accounted for most of the costs in all five countries (80, 100, 99, 98, and 93 %, respectively)., Conclusions: MPS patients experience substantial loss of HRQOL and their families take a remarkable part in their care. Although utilization of health and social care resources varies significantly across countries, MPS incurs considerable societal costs in all the countries studied.

Objective: The aim of this study was to determine the economic burden from a societal perspective and health-related quality of life (HRQOL) of patients with Prader-Willi syndrome (PWS) in Europe., Methods: We conducted a cross-sectional study of patients with PWS from Spain, Bulgaria, Hungary, Germany, Italy, the UK, Sweden and France. Data on demographic characteristics, healthcare resource utilisation, informal care, labour productivity losses and HRQOL were collected from questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) questionnaire., Results: A total of 261 patients completed the questionnaire. The average annual costs ranged from € 3937 to € 67,484 between countries; the reference year for unit prices was 2012. Direct healthcare costs ranged from € 311 to € 18,760, direct non-healthcare costs ranged from € 1269 to € 44,035, and loss of labour productivity ranged from € 0 to € 2255. Costs were also shown to differ between children and adults. The mean EQ-5D index score for adult PWS patients ranged between 0.40 and 0.81 and the mean EQ-5D visual analogue scale score ranged between 51.25 and 90.00., Conclusion: The main strengths of this study lie in our bottom-up approach to costing and in the evaluation of PWS patients from a broad societal perspective. This type of analysis is very scarce in the international literature on rare diseases in comparison with other illnesses. We conclude that PWS patients incur considerable societal costs and experience substantial deterioration in HRQOL.

Objective: The aim of this study was to determine the economic burden from a societal perspective and the health-related quality of life (HRQOL) of patients with systemic sclerosis (SSc; scleroderma) in Europe., Methods: We conducted a cross-sectional study of patients with SSc (involving both localised and systemic sclerosis) from Germany, Italy, Spain, France, the UK, Hungary and Sweden. Data on demographic characteristics, healthcare resource utilisation, informal care, labour productivity losses and HRQOL were collected from the questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) questionnaire., Results: A total of 589 patients completed the questionnaire. The rate of patients with localised scleroderma, limited cutan and diffuse cutan SSc were 28, 68 and 4 %, respectively. Average annual costs varied from country to country and ranged from € 4607 to € 30,797 (reference year: 2012). Estimated direct healthcare costs ranged from € 1413 to € 17,300; direct non-healthcare costs ranged from € 1875 to € 4684 and labour productivity losses ranged from € 1701 to € 14,444. The mean EQ-5D index score for adult SSc patients varied from 0.49 to 0.75 and the mean EQ-5D visual analogue scale score was between 58.72 and 65.86., Conclusion: The main strengths of this study lie in our bottom-up approach to costing and our evaluation of SSs patients from a broad societal perspective. This type of analysis is very unusual in the international literature on rare diseases in comparison with other illnesses. We concluded that SSc patients incur considerable societal costs and experience substantial deterioration in HRQOL.